A pivotal trial of Omeros Corp.'s narsoplimab, an investigational therapy for an occasional but serious complication of hematopoietic stem cell transplant (HSCT), found that just one dose was enough to deliver a beneficial effect in more than half the study's high-risk participants, meeting the study's primary endpoint.
Protagonist Therapeutics Inc. stock (NASDAQ:PTGX) nosedived 47% on Tuesday after the company released preliminary results from its phase II open-label study of PTG-300, an injectable hepcidin mimetic to treat patients with transfusion-dependent beta-thalassemia.
With nearly 5,000 abstracts on tap for this December's American Society of Hematology (ASH) meeting in Orlando, Fla., organizers of the annual conference have highlighted a multitude of new advances in the event's programming.
Less than two weeks after giving the go-ahead to Novartis AG for Adakveo (crizanlizumab) to reduce the frequency of vaso-occlusive crises (VOCs) in adult and pediatric patients ages 16 and older with sickle cell disease (SCD), the FDA cleared – well ahead of its Feb. 26, 2020, PDUFA date – Oxbryta (voxelotor), from Global Blood Therapeutics Inc. (GBT), for SCD in adults and pediatric patients ages 12 and up.
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